Recent Updates
Recently added Catalysts

Deflazacort

Phase 1

Renal Impairment | Small molecule | Nephrology |PTC Therapeutics, Inc.|Last Updated: Aug 18, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment16
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort PharmacokineticsPHASE1 COMPLETED 16Dec 1, 2014Feb 1, 2015Aug 18, 20172 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Renal impairment on the pharmacokinetics (PK) of deflazacort in subjects with end-stage renal disease including the area under the plasma concentration time curve, from time 0 to the last measurable non-zero concentration.
1 day
Secondary Endpoints
Safety and tolerability of one dose of deflazacort in subjects with end stage renal disease as measured by capturing occurrence of adverse events.
1 day
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
Renal ImpairmentEXPERIMENTALEight (8) subjects with ESRD on HD will receive one 18 mg dose of deflazacort.
Healthy VolunteersEXPERIMENTALEight (8) healthy subjects with estimated creatinine clearance (CLcr) ≥ 90 mL/min. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the ESRD cohort. Subjects will receive one 18 mg dose of deflazacort.
Interventions
NameTypeDescription
DeflazacortDRUGDeflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite 21-desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: * Continuous non-smokers or moderate smokers * For a female of non-childbearing potential: must have undergone a sterilization procedures or be postmenopausal with amenorrhea for at least 1 year prior to the first dose and FSH serum levels consistent with postmenopausal status *...

Countries:United States
Unlock Eligibility Criteria